Session Chair(s)

Adora Ndu, JD, PharmD

Executive Director, Global Regulatory Policy, Research and Engagement

BioMarin Pharmaceutical Inc., United States

This session will explore the challenges and opportunities related to patient-focused clinical outcome assessment (COA) endpoint selection, development and implementation in rare and ultra-rare disease drug development. Panelists from industry, the FDA and instrument development experts will provide insight into the identification and implementation of fit-for-purpose COAs in rare disease trials, including challenges specific to endpoint strategy development and clinical trial design. Relevant case studies will be highlighted and special considerations discussed.

Learning Objective : Describe opportunities and challenges in rare disease drug development; Identify how challenges in rare disease clinical trials may be addressed and overcome; Determine how innovators and patient advocates can work with FDA to promote innovation in this space.

Panelist

Kate Delaney

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